New FRMP gene targets: potential for new treatments in FXS/ASD

Posted by & filed under Part 28: NEUROGENETICS.

Fragile X syndrome (FXS) is typically caused by the loss of fragile X mental retardation 1 (FMR1) expression, which codes for the RNA-binding protein FMRP. Ascano et al. reported the identification of RNA-recognition elements for FMRP, in addition to its target messenger RNAs. Many of FMRP gene targets discovered are involved in brain function and autism spectrum disorder (ASD). It appears that FMRP affects their protein levels in human cell culture, mouse ovaries and human brain. These targets are also dysregulated in Fmr1 -/- mouse ovaries showing signs of premature follicular overdevelopment. Ascano et al. concluded that their ranked list of genes provide the basis for the pursuit of new therapeutic targets for FXS and ASD.

FMRP targets distinct mRNA sequence elements to regulate protein expression. Ascano et al. Nature. 2012 Dec 20;492(7429):382-6. PMID: 23235829

posted by Yannis Trakadis, MD

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